Published on 13 May 2022 on Zacks via Yahoo Finance
Editas Medicine, Inc. EDIT announced that the FDA has granted Orphan Drug designation to its investigational gene-editing medicine, EDIT-301, for the treatment of beta thalassemia, a devastating disease that leads to severe anemia, organ failure and premature death.
The Orphan Drug designation is granted by the FDA to a drug or biologic intended to treat a rare disease or condition, which generally includes one that affects fewer than 200,000 individuals in the United States. The designation also includes incentives, including financial aid for clinical testing, and seven-year marketing exclusivity in the event of regulatory approval.
Per the company, the Orphan Drug tag for EDIT-301 for treating beta thalassemia underlines the urgent need for new treatment options for the given patient population.